CA2645120C - Treatment of cns conditions - Google Patents
Treatment of cns conditions Download PDFInfo
- Publication number
- CA2645120C CA2645120C CA2645120A CA2645120A CA2645120C CA 2645120 C CA2645120 C CA 2645120C CA 2645120 A CA2645120 A CA 2645120A CA 2645120 A CA2645120 A CA 2645120A CA 2645120 C CA2645120 C CA 2645120C
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- XXYIANZGUOSQHY-XLPZGREQSA-N thymidine 3'-monophosphate Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](OP(O)(O)=O)C1 XXYIANZGUOSQHY-XLPZGREQSA-N 0.000 description 1
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- QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 1
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Classifications
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
- A61K2039/541—Mucosal route
- A61K2039/543—Mucosal route intranasal
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
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- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
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- Neurology (AREA)
- Neurosurgery (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
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- Physics & Mathematics (AREA)
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- Plant Pathology (AREA)
- Psychology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
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| GBGB0605337.5A GB0605337D0 (en) | 2006-03-17 | 2006-03-17 | Treatment of CNS conditions |
| GB0605337.5 | 2006-03-17 | ||
| PCT/GB2007/050128 WO2007107789A2 (en) | 2006-03-17 | 2007-03-16 | Treatment of cns conditions |
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| CA2645120A1 CA2645120A1 (en) | 2007-09-27 |
| CA2645120C true CA2645120C (en) | 2015-02-24 |
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| CA2645120A Active CA2645120C (en) | 2006-03-17 | 2007-03-16 | Treatment of cns conditions |
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| US (1) | US8871729B2 (ru) |
| EP (1) | EP2004823B1 (ru) |
| JP (2) | JP5781722B2 (ru) |
| KR (2) | KR101605144B1 (ru) |
| CN (1) | CN101448944B (ru) |
| AU (1) | AU2007228570B2 (ru) |
| CA (1) | CA2645120C (ru) |
| ES (1) | ES2582649T3 (ru) |
| GB (1) | GB0605337D0 (ru) |
| HK (1) | HK1126812A1 (ru) |
| IL (1) | IL194114A (ru) |
| MX (1) | MX2008011731A (ru) |
| RU (1) | RU2426544C2 (ru) |
| WO (1) | WO2007107789A2 (ru) |
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| US20080249058A1 (en) * | 2007-04-05 | 2008-10-09 | Erik Roberson | Agents that reduce neuronal overexcitation |
| WO2010080452A2 (en) | 2008-12-18 | 2010-07-15 | Quark Pharmaceuticals, Inc. | siRNA COMPOUNDS AND METHODS OF USE THEREOF |
| CN102625809B (zh) * | 2009-09-11 | 2015-06-24 | Isis制药公司 | 亨廷顿表达的调节 |
| JP2013511990A (ja) | 2009-11-26 | 2013-04-11 | クォーク ファーマシューティカルズ インコーポレーティッド | 末端置換を含むｓｉＲＮＡ化合物 |
| US20110135613A1 (en) * | 2009-12-03 | 2011-06-09 | The J. David Gladstone Institutes | Methods for treating apolipoprotein e4-associated disorders |
| WO2011072091A1 (en) | 2009-12-09 | 2011-06-16 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the cns |
| JP6081798B2 (ja) * | 2010-02-26 | 2017-02-15 | メモリアル スローン−ケタリング キャンサー センター | ｍｉＲＮＡに関連する癌を検出および処置するための方法および組成物およびｍｉＲＮＡインヒビターおよび標的 |
| ES2638309T3 (es) | 2010-04-19 | 2017-10-19 | Nlife Therapeutics S.L. | Composiciones y métodos para la distribución selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas |
| EP2380595A1 (en) | 2010-04-19 | 2011-10-26 | Nlife Therapeutics S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
| ES2663009T3 (es) | 2010-10-29 | 2018-04-10 | Sirna Therapeutics, Inc. | Inhibición de la expresión génica mediada por interferencia por ARN utilizando ácidos nucleicos de interferencia cortos (ANic) |
| WO2012078536A2 (en) | 2010-12-06 | 2012-06-14 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising positional modifications |
| US9949923B2 (en) * | 2011-03-15 | 2018-04-24 | Optinose As | Nasal delivery |
| EP2766495B1 (en) * | 2011-10-14 | 2018-04-04 | Centre National De La Recherche Scientifique | Method of diagnosis, prognostic or treatment of neurodegenerative diseases |
| WO2013134777A1 (en) * | 2012-03-09 | 2013-09-12 | Northeastern University | Methods for delivery to the central nervous system of nucleic acid nanoparticles to treat central nervous system disorders |
| CA2866392C (en) | 2012-03-30 | 2023-10-24 | Washington University | Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| US9611473B2 (en) | 2012-09-12 | 2017-04-04 | Quark Pharmaceuticals, Inc. | Double-stranded nucleic acid compounds |
| CA2890112A1 (en) | 2012-10-26 | 2014-05-01 | Nlife Therapeutics, S.L. | Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
| RU2745324C2 (ru) | 2013-03-14 | 2021-03-23 | Ионис Фармасьютикалз, Инк. | Композиции и способы модулирования экспрессии tau |
| JP2016523980A (ja) | 2013-07-11 | 2016-08-12 | ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク | タウ発現を抑制するマイクロｒｎａ |
| TWI772856B (zh) | 2013-07-19 | 2022-08-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| EP3027223A1 (en) | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Methods of use of sphingolipid polyalkylamine oligonucleotide compounds |
| WO2015089375A1 (en) * | 2013-12-13 | 2015-06-18 | The General Hospital Corporation | Soluble high molecular weight (hmw) tau species and applications thereof |
| WO2016151523A1 (en) * | 2015-03-25 | 2016-09-29 | Università Degli Studi Di Trento | Rna interference mediated therapy for neurodegenerative diseases |
| JOP20200228A1 (ar) * | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
| JOP20190065A1 (ar) | 2016-09-29 | 2019-03-28 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن tau |
| WO2019070894A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMIAL DISORDERS |
| EP3692151A4 (en) | 2017-10-03 | 2021-07-14 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
| MX2020009532A (es) * | 2018-03-13 | 2020-10-05 | Janssen Pharmaceutica Nv | Oligonucleotidos modificados para uso en el tratamiento de tauopatias. |
| CN115552006A (zh) * | 2020-03-18 | 2022-12-30 | 马萨诸塞大学 | 用于mapt调节的寡核苷酸 |
| CN113817728A (zh) * | 2020-06-19 | 2021-12-21 | 四川大学华西医院 | 一种有效干扰Tau蛋白表达的重组慢病毒及其应用 |
| TW202400792A (zh) * | 2022-05-12 | 2024-01-01 | 美商戴瑟納製藥股份有限公司 | 用於抑制ｍａｐｔ表現之組合物及方法 |
| WO2024145474A2 (en) * | 2022-12-29 | 2024-07-04 | Voyager Therapeutics, Inc. | Compositions and methods for regulating mapt |
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| CA2116460A1 (en) | 1993-03-02 | 1994-09-03 | Mitsubishi Chemical Corporation | Preventive or therapeutic agents for alzheimer's disease, a screening method of alzheimer's disease and tau-protein kinase i originated from human being |
| US6410046B1 (en) * | 1996-11-19 | 2002-06-25 | Intrabrain International Nv | Administering pharmaceuticals to the mammalian central nervous system |
| WO2005003350A2 (en) | 2003-06-27 | 2005-01-13 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| WO2002086105A1 (en) * | 2001-04-20 | 2002-10-31 | Chiron Corporation | Delivery of polynucleotide agents to the central nervous sysstem |
| US20050159376A1 (en) | 2002-02-20 | 2005-07-21 | Slrna Therapeutics, Inc. | RNA interference mediated inhibition 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA) |
| IL143379A (en) | 2001-05-24 | 2013-11-28 | Yissum Res Dev Co | Oligonucleotide against human ache isoform r and its uses |
| EP1432724A4 (en) | 2002-02-20 | 2006-02-01 | Sirna Therapeutics Inc | RNA inhibition mediated inhibition of MAP KINASE GENES |
| GB2406568B (en) | 2002-02-20 | 2005-09-28 | Sirna Therapeutics Inc | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| AU2003224087B2 (en) | 2002-04-18 | 2009-03-05 | Opko Pharmaceuticals, Llc. | Means and methods for the specific inhibition of genes in cells and tissue of the CNS and/or eye |
| US20040254146A1 (en) | 2002-05-21 | 2004-12-16 | Nastech Pharmaceutical Company Inc. | Carboxylate salts of galantamine and their pharmaceutical use |
| US20030225031A1 (en) | 2002-05-21 | 2003-12-04 | Quay Steven C. | Administration of acetylcholinesterase inhibitors to the cerebral spinal fluid |
| US20050106731A1 (en) | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US20040241854A1 (en) | 2002-08-05 | 2004-12-02 | Davidson Beverly L. | siRNA-mediated gene silencing |
| AU2003282877B9 (en) | 2002-09-25 | 2011-05-12 | University Of Massachusetts | In Vivo gene silencing by chemically modified and stable siRNA |
| WO2004028471A2 (en) | 2002-09-28 | 2004-04-08 | Massachusetts Institute Of Technology | Influenza therapeutic |
| ES2334125T3 (es) * | 2002-11-04 | 2010-03-05 | University Of Massachusetts | Interferencia de arn especifico de alelos. |
| US7829694B2 (en) | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| WO2005004794A2 (en) * | 2003-06-09 | 2005-01-20 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
| WO2005044976A2 (en) | 2003-06-20 | 2005-05-19 | Isis Pharmaceuticals, Inc. | Oligomeric compounds for use in gene modulation |
| WO2005014815A1 (en) * | 2003-08-08 | 2005-02-17 | President And Fellows Of Harvard College | siRNA BASED METHODS FOR TREATING ALZHEIMER’S DISEASE |
| WO2005045037A2 (en) | 2003-10-23 | 2005-05-19 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF 5-ALPHA REDUCTASE AND ANDROGEN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| WO2005105995A2 (en) | 2004-04-14 | 2005-11-10 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| US20050265927A1 (en) | 2004-05-17 | 2005-12-01 | Yale University | Intranasal delivery of nucleic acid molecules |
| WO2006041922A2 (en) | 2004-10-08 | 2006-04-20 | Dara Biosciences, Inc. | Agents and methods for administration to the central nervous system |
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Also Published As
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| CN101448944A (zh) | 2009-06-03 |
| US20090176728A1 (en) | 2009-07-09 |
| EP2004823A2 (en) | 2008-12-24 |
| IL194114A (en) | 2012-06-28 |
| AU2007228570A8 (en) | 2008-10-23 |
| RU2426544C2 (ru) | 2011-08-20 |
| IL194114A0 (en) | 2011-08-01 |
| KR101669944B1 (ko) | 2016-10-28 |
| GB0605337D0 (en) | 2006-04-26 |
| JP5781722B2 (ja) | 2015-09-24 |
| ES2582649T3 (es) | 2016-09-14 |
| CA2645120A1 (en) | 2007-09-27 |
| HK1126812A1 (zh) | 2009-09-11 |
| KR20080111063A (ko) | 2008-12-22 |
| AU2007228570B2 (en) | 2013-09-19 |
| JP2013234197A (ja) | 2013-11-21 |
| WO2007107789A3 (en) | 2008-05-08 |
| EP2004823B1 (en) | 2016-05-25 |
| JP5923466B2 (ja) | 2016-05-24 |
| JP2009530257A (ja) | 2009-08-27 |
| US8871729B2 (en) | 2014-10-28 |
| RU2008141162A (ru) | 2010-04-27 |
| AU2007228570A1 (en) | 2007-09-27 |
| WO2007107789A2 (en) | 2007-09-27 |
| KR101605144B1 (ko) | 2016-03-21 |
| MX2008011731A (es) | 2009-03-05 |
| KR20140076634A (ko) | 2014-06-20 |
| CN101448944B (zh) | 2014-02-12 |
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